Ivacaftor withdrawal syndrome in cystic fibrosis patients with the G551D mutation

Aaron T. Trimble, Scott H. Donaldson

Research output: Contribution to journalArticlepeer-review

16 Scopus citations

Abstract

Ivacaftor use can lead to dramatic health improvements in cystic fibrosis (CF) patients with gating mutations. Here, we report five instances of dramatic clinical decline following withdrawal of ivacaftor in three individuals with the G551D-CFTR mutation. In each case, the patient's lung function and symptoms rapidly deteriorated after cessation of treatment. Awareness of this phenomenon should inform both clinical practices as well as the design of future clinical trials of highly active CFTR modulators.

Original languageEnglish (US)
Pages (from-to)e13-e16
JournalJournal of Cystic Fibrosis
Volume17
Issue number2
DOIs
StatePublished - Mar 2018
Externally publishedYes

Keywords

  • Acute pulmonary exacerbation
  • CFTR modulator
  • Cystic fibrosis
  • G551D
  • Ivacaftor

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Pulmonary and Respiratory Medicine

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